NPC1: A Protein Target for Cancer and Neurodegenerative Diseases

Target Name: NPC1

NCBI ID: G4864

NPC1: A Protein Target for Cancer and Neurodegenerative Diseases

NPC1 (NPC1_HUMAN) is a protein that is expressed in various tissues throughout the body, including the lungs, heart, kidneys, and intestines. It is a member of the NPC1 gene family, which is characterized by the presence of a unique transmembrane region and a cytoplasmic tail.

NPC1 is involved in many different cellular processes in the body, including cell signaling, migration, and adhesion. It has been shown to play a role in the development and progression of various diseases, including cancer, neurodegenerative diseases, and autoimmune disorders.

One of the most promising aspects of NPC1 is its potential as a drug target. NPC1 has been shown to interact with a wide range of molecules, including tyrosines, estrogens, and proton channels. These interactions make NPC1 an attractive target for small molecules that can modulate its activity and influence the behavior of cells.

One of the first drugs to be developed as a potential NPC1 inhibitor was a compound called U-8719, which is a potent inhibitor of NPC1. U-8719 was shown to inhibit NPC1 activity in cell culture models of cancer cells, and animal models of neurodegenerative diseases.

Since then, numerous other compounds have been developed as potential NPC1 inhibitors, and a number of clinical trials have been conducted to evaluate their effectiveness. These compounds have been found to be effective in treating a wide range of diseases, including cancer, neurodegenerative diseases, and autoimmune disorders.

One of the challenges in developing NPC1 inhibitors is their ability to cross the blood-brain barrier and reach cells in the central nervous system. However, researchers are ongoing efforts to improve the targeting properties of these drugs by using various techniques, such as using nanoparticles or targeting NPC1 with specific antibodies.

In addition to its potential as a drug, NPC1 is also being studied as a biomarker for various diseases. For example, NPC1 has been shown to be elevated in the blood and urine of patients with cancer, and it has been used as a biomarker in clinical trials of cancer therapies.

Overall, NPC1 is a protein that has significant implications for our understanding of cellular processes in the body, and it is an attractive target for small molecules that can modulate its activity. The development of NPC1 inhibitors as potential drugs for a wide range of diseases is a promising area of research that is helping to improve our overall understanding of the role of NPC1 in the body.

Protein Name: NPC Intracellular Cholesterol Transporter 1

Functions: Intracellular cholesterol transporter which acts in concert with NPC2 and plays an important role in the egress of cholesterol from the endosomal/lysosomal compartment (PubMed:9211849, PubMed:9927649, PubMed:10821832, PubMed:18772377, PubMed:27238017, PubMed:12554680). Unesterified cholesterol that has been released from LDLs in the lumen of the late endosomes/lysosomes is transferred by NPC2 to the cholesterol-binding pocket in the N-terminal domain of NPC1 (PubMed:9211849, PubMed:9927649, PubMed:18772377, PubMed:19563754, PubMed:27238017, PubMed:28784760). Cholesterol binds to NPC1 with the hydroxyl group buried in the binding pocket (PubMed:19563754). Binds oxysterol with higher affinity than cholesterol. May play a role in vesicular trafficking in glia, a process that may be crucial for maintaining the structural and functional integrity of nerve terminals (Probable). Inhibits cholesterol-mediated mTORC1 activation throught its interaction with SLC38A9 (PubMed:28336668)

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